University Medical Center CF, Kerey-Zhanibek Handar St. 5/1, Z05P3Y4, Astana, Kazakhstan.
Astana Medical University, Beybitshilik St. 49A, Z10K9D9, Astana, Kazakhstan.
Mol Neurobiol. 2023 Aug;60(8):4324-4335. doi: 10.1007/s12035-023-03346-3. Epub 2023 Apr 24.
In Kazakhstan, there is insufficient data on genetic epilepsy, which has its own clinical and management implications. Thus, this study aimed to use whole genome sequencing to identify and evaluate genetic variants and genetic structure of early onset epilepsy in the Kazakhstani pediatric population. In this study, for the first time in Kazakhstan, whole genome sequencing was carried out among epilepsy diagnosed children. The study involved 20 pediatric patients with early onset epilepsy and no established cause of the disease during the July-December, 2021. The average age at enrolment was 34.5 months, with a mean age at seizure onset of 6 months. Six patients (30%) were male, and 7 were familial cases. We identified pathogenic and likely pathogenic variants in 14 (70%) cases, among them, 6 novel disease gene variants (KCNQ2, CASK, WWOX, MT-CO3, GRIN2D, and SLC12A5). Other genes associated with the disease were SCN1A (x2), SLC2A1, ARX, CACNA1B, PCDH19, KCNT1, and CHRNA2. Identification of the genetic causes in 70% of cases confirms the general structure of the etiology of early onset epilepsy and the necessity of using NGS in diagnostics. Moreover, the study describes new genotype-phenotypic correlations in genetic epilepsy. Despite certain limitations of the study, it can be concluded that the genetic etiology of pediatric epilepsy in Kazakhstan is very broad and requires further research.
在哈萨克斯坦,关于遗传性癫痫的数据不足,这对其临床和管理具有重要意义。因此,本研究旨在利用全基因组测序来识别和评估哈萨克斯坦儿科人群中早发性癫痫的遗传变异和遗传结构。在这项研究中,首次在哈萨克斯坦对确诊患有癫痫的儿童进行了全基因组测序。该研究纳入了 20 名患有早发性癫痫且病因不明的儿科患者,入组时间为 2021 年 7 月至 12 月。平均入组年龄为 34.5 个月,癫痫发作的平均年龄为 6 个月。6 名患者(30%)为男性,7 名为家族性病例。我们在 14 例(70%)患者中发现了致病性和可能致病性变异,其中包括 6 种新的疾病基因变异(KCNQ2、CASK、WWOX、MT-CO3、GRIN2D 和 SLC12A5)。其他与疾病相关的基因有 SCN1A(x2)、SLC2A1、ARX、CACNA1B、PCDH19、KCNT1 和 CHRNA2。在 70%的病例中确定了遗传病因,这证实了早发性癫痫病因的总体结构,以及在诊断中使用 NGS 的必要性。此外,该研究描述了遗传性癫痫中新的基因型-表型相关性。尽管研究存在一定局限性,但可以得出结论,哈萨克斯坦儿科癫痫的遗传病因非常广泛,需要进一步研究。